The OneGene Program

The OneGene Program is a team of highly trained and dedicated people who help ensure that every family has the support they need during their treatment journey and beyond. Their work starts the moment the ZOLGENSMA® (onasemnogene abeparvovec-xioi) Prescription Form and Patient Consent Form are received. From that point forward, the team works closely with your family to do everything from answering questions to being a reassuring voice to speak to when things might feel overwhelming.

Contact the OneGene Program at 855-441-GENE (4363), Monday-Friday (8 AM-8 PM), to learn more.

The OneGene Program

The OneGene Program is a team of highly trained and dedicated people who help ensure that every family has the support they need during their treatment journey and beyond. Their work starts the moment the ZOLGENSMA® (onasemnogene abeparvovec-xioi) Prescription Form and Patient Consent Form are received. From that point forward, the team works closely with your family to do everything from answering questions to being a reassuring voice to speak to when things might feel overwhelming.

Contact the OneGene Program at 855-441-GENE (4363), Monday-Friday (8 AM-8 PM), to learn more.

Support available to every family includes:

A patient resource manager icon

Patient Resource Manager

Your Patient Resource Manager is your main point of contact on behalf of AveXis. This person provides expert guidance and emotional support at every step of your treatment journey and answers questions about ZOLGENSMA.

A field reimbursement manager iconA case coordinator icon

Other members of your support team

A Field Reimbursement Manager helps your doctor navigate insurance requirements and authorizations. Other support includes following your child’s prescription through insurance approvals, the pharmacy process, and shipment journey to ensure it is delivered to the right place at the right time.

A Case Coordinator acts as a go-between to connect you to resources you may need and assists doctors with insurance processes. Other support includes verification of insurance benefits and coordination of financial assistance programs for eligible patients.

I am passionate about helping families living with SMA because I care deeply about supporting families as they face the realities of a rare disease diagnosis. I want to help ease your burden in any way I can. I am dedicated to helping you as we face your challenges together. Karen, Patient Resource Manager

A Patient Resource Manager can be invaluable to families. Caregivers need help staying organized, and they need help knowing what to do. Patient Resource Managers can help them with that.

Milan, father of Evelyn
Evelyn, treated at ~2 months and pictured at 3½ years, has SMA Type 1.

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

  • ZOLGENSMA can cause acute serious liver injury. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive ZOLGENSMA.
  • Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.

Important Safety Information

What is the most important information I should know about ZOLGENSMA?

  • ZOLGENSMA can cause acute serious liver injury. Liver enzymes could become elevated and may reflect acute serious liver injury in children who receive ZOLGENSMA.
  • Patients will receive an oral corticosteroid before and after infusion with ZOLGENSMA and will undergo regular blood tests to monitor liver function.
  • Contact the patient’s doctor immediately if the patient’s skin and/or whites of the eyes appear yellowish, or if the patient misses a dose of the corticosteroid or vomits it up.

What should I watch for before and after infusion with ZOLGENSMA?

  • Viral respiratory infections before or after ZOLGENSMA infusion can lead to more serious complications. Contact the patient’s doctor immediately if you see signs of a possible viral respiratory infection such as coughing, wheezing, sneezing, runny nose, sore throat, or fever.
  • Decreased platelet counts could occur following infusion with ZOLGENSMA. Seek immediate medical attention if a patient experiences unexpected bleeding or bruising.

What do I need to know about vaccinations and ZOLGENSMA?

  • Talk with the patient’s doctor to decide if adjustments to the vaccination schedule are needed to accommodate treatment with a corticosteroid.
  • Protection against respiratory syncytial virus (RSV) is recommended.

Do I need to take precautions with the patient’s bodily waste?
Temporarily, small amounts of ZOLGENSMA may be found in the patient’s stool. Use good hand hygiene when coming into direct contact with bodily waste for 1 month after infusion with ZOLGENSMA. Disposable diapers should be sealed in disposable trash bags and thrown out with regular trash.

What are the possible or likely side effects of ZOLGENSMA?
The most common side effects that occurred in patients treated with ZOLGENSMA were elevated liver enzymes and vomiting.

Indication

What is ZOLGENSMA?
ZOLGENSMA is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). ZOLGENSMA is given as a one-time infusion into the vein. ZOLGENSMA was not evaluated in patients with advanced SMA.

The safety information provided here is not comprehensive. Talk to the patient’s doctor about any side effects that bother the patient or that don’t go away.

You are encouraged to report suspected side effects by contacting the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch, or AveXis at 833-828-3947.

Please see the Full Prescribing Information.